NCT07246941

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

A Phase I, 2-Part Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-ascending Doses of Intrathecally Administered RG6496 in a Randomized, Placebo-controlled, Investigator/Participant-blind Study With an Open-label Extension in Huntington's Disease Gene Expansion Carriers

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 25 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label extension (OLE)\].

Conditions

• Huntington's Disease

Interventions

Type	Name	Description
DRUG	RG6496	RG6496 will be administered as per the schedule specified in the respective arms.
DRUG	Placebo	RG6496 matching placebo will be administered as per the schedule specified in placebo arm.

Timeline

• Start date: 2025-11-19
• Primary completion: 2027-12-11
• Completion: 2029-03-05
• First posted: 2025-11-24
• Last updated: 2026-03-30

Locations

3 sites across 3 countries: Argentina, Australia, New Zealand